Durability and Immunogenicity of Inhaled SP-101, A Novel AAV2.5T Gene Therapy for Cystic Fibrosis

Time: 11:30 am
day: Day One AM


• Understanding the unique durability challenges relating to delivering gene therapies via inhalation to the lung

• Exploring a novel AAV capsid (AAV2.5T) for inhaled delivery of a CFTR minigene (hCFTRdR) for the treatment of Cystic Fibrosis

• Assessing data to understand the durability of hCFTRdR transgene expression and associated immunogenicity